Study on Mannan Binding Lectin (MBL) Substitution in MBL-Deficient Children With Chemotherapy-Induced Neutropenia

NCT00138736 | Completed Phase 2

• 1 other identifier: KB2003.02
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 2

Brief Summary

The pharmacokinetics, and clinical and biological effects of MBL replacement therapy in MBL-deficient children during chemotherapy-induced neutropenia were studied.

Conditions

MBL-Deficient; Neutropenia

Keywords

MBL-deficient; chemotherapy; neutropenia; chemotherapy-induced neutropenia

Outcome Measures

Primary Outcomes (1)

• pharmacokinetics of MBL

  until the patient's absolute neutrophil count (ANC) is above 500/uL blood.

Secondary Outcomes (6)

• days of fever

  until the patient's absolute neutrophil count (ANC) is above 500/uL blood.

• days of hospital admission

  until the patient's absolute neutrophil count (ANC) is above 500/uL blood.

• use of antibiotics or antifungal medication

  until the patient's absolute neutrophil count (ANC) is above 500/uL blood.

• number and type of infections

  until the patient's absolute neutrophil count (ANC) is above 500/uL blood.

• MBL-dependent opsonizing capacity in vitro

  until the patient's absolute neutrophil count (ANC) is above 500/uL blood.

Study Arms (1)

A

EXPERIMENTAL

MBL until the patient's absolute neutrophil count (ANC) is above 500/microL blood.

Drug: Mannan Binding Lectin (MBL); Drug: Mannan Binding Lectin

Interventions

Mannan Binding Lectin (MBL) DRUG

A

Mannan Binding Lectin DRUG

MBL dose at a twice weekly dose interval (3 or 4 days): 0.2 mg/kg, for a 3-day interval; 0.3 mg/kg, for a 4-day interval

Also known as: MBL SSI

A

Eligibility Criteria

• Age: Up to 12 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Children ages 0 - 12 years, during chemotherapy, and expected to become neutropenic
• MBL deficiency by genotype or phenotype (\< 100 ng/ml)
• Informed consent and assent of patient and/or legal representative

You may not qualify if:

• Inability or unwillingness to comply with the protocol or likely inability to complete the study period
• Known allergic reactions to MBL and other human plasma products
• Participation in other investigational drug studies within the last month
• Clinically relevant abnormalities in: serum immunoglobulins IgG, IgA, IgM; blood counts; complement factors measured by AP50, CH50; urine protein and cell counts; serum creatinine and liver enzymes, as routinely determined for regular patient care.

Sponsors & Collaborators

• Prothya Biosolutions: lead
• Landsteiner Foundation for Blood Transfusion: collaborator

Study Sites (2)

Academic Medical Centre

Amsterdam, 1105 AZ, Netherlands

Location

Erasmus Medical Centre

Rotterdam, 3015 GD, Netherlands

Location

MeSH Terms

Conditions

Neutropenia

Interventions

Mannose-Binding Lectin

Condition Hierarchy (Ancestors)

Agranulocytosis; Leukopenia; Cytopenia; Hematologic Diseases; Hemic and Lymphatic Diseases; Leukocyte Disorders

Intervention Hierarchy (Ancestors)

Collectins; Lectins, C-Type; Lectins; Proteins; Amino Acids, Peptides, and Proteins; Mannose-Binding Lectins

Study Officials

• T W Kuijpers, MD, PdD

  Dept. of Paediatric Immunology, AMC, Amsterdam, The Netherlands

  PRINCIPAL INVESTIGATOR

• H N Caron

  Dept of Paediatric Oncology, AMC, Amsterdam, The Netherlands

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: PREVENTION
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY

Study Record Dates

• First Submitted: August 29, 2005
• First Posted: August 30, 2005
• Study Start: April 1, 2004
• Study Completion: October 1, 2006
• Last Updated: August 30, 2007

Record last verified: 2007-08

Locations

NL (2)